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Molecular Profile VHL inact mut
Therapy Belzutifan
Indication/Tumor Type renal cell carcinoma
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
VHL inact mut renal cell carcinoma sensitive Belzutifan Guideline Actionable Welireg (belzutifan) is included in guidelines for patients with Von Hippel-Lindau disease-associated renal cell carcinoma that do not require immediate surgery (NCCN.org). detail...
VHL inact mut renal cell carcinoma sensitive Belzutifan Guideline Actionable Welireg (belzutifan) is included in guidelines for patients with localized Von Hippel-Lindau disease-associated renal cell carcinoma harboring germline VHL mutations (PMID: 38788900; ESMO.org). detail... 38788900
VHL inact mut renal cell carcinoma sensitive Belzutifan FDA approved Actionable In a Phase II trial that supported FDA approval, Welireg (belzutifan) treatment was safe and resulted in an objective response in 49% (30/61, 30 partial responses) and stable disease in 49% of patients with Von Hippel-Lindau disease-associated localized renal cell carcinoma harboring germline VHL mutations, 94% of patients were progression-free at 24 months and median duration of response was not reached with a median follow-up of 21.8 months (PMID: 34818478; NCT03401788). 34818478 detail...
PubMed Id Reference Title Details
NCCN.org Full reference...
ESMO Clinical Practice Guidelines Full reference...
Welireg (belzutifan) FDA Drug Label Full reference...
(38788900) Renal cell carcinoma: ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up. Full reference...
(34818478) Belzutifan for Renal Cell Carcinoma in von Hippel-Lindau Disease. Full reference...